Patient resources

Clinical research studies help scientists and doctors explore whether a medical strategy, drug, or device is safe and effective for humans. Participation in clinical research studies is voluntary, and participants can stop at any time.

Xerostomia (dry mouth) is a condition in which a person’s parotid salivary glands do not produce enough saliva. It can be caused by damage from radiation therapy for conditions like head and neck cancer. Currently, there is no cure for xerostomia. Most therapies focus on managing the symptoms.

Parotid Glands
The parotid glands are under the skin in front of each ear. Damage to these glands reduces the amount of saliva they make. This can lead to other problems in the mouth like yeast infections, excessive tooth decay, and sores.

The AQUAx2 study is a clinical research study for people with dry mouth (xerostomia) caused by radiation therapy from previous head and neck cancer. The purpose of this study is to assess the efficacy and safety of a new investigational gene transfer study drug, called AAV2-hAQP1, at helping with dry mouth resulting from radiation damage to the parotid salivary glands.

The purpose of this study is to see if the investigational gene therapy, AAV2-hAQP1 (study drug), has an effect on the amount of saliva produced and on dry mouth symptoms, and to see if it is safe and well-tolerated in people with dry mouth as a result of receiving radiation therapy for head and neck cancer.

If you qualify for the study, you will be randomly assigned to one of three treatment groups via a process called randomization (decided by chance). Two of these three groups will receive the active gene therapy (one of two different strengths of the study drug) and the third will receive a placebo (an inactive solution that does not contain the study drug). If you receive the placebo in this study, you will be offered the active gene therapy in a long term follow-up study.

The study drug is a new, investigational gene transfer therapy drug. It is given as a gene injection through the parotid gland. No anesthesia is needed. The study drug delivers a gene designed to make a specific protein. This study will see if this protein helps the parotid gland make more saliva. The study drug is investigational, which means it is still being studied. Regulatory authorities like the US Food and Drug Administration (FDA) and Health Canada do not currently allow it to be sold as therapy for xerostomia.

Study participants must meet certain criteria* to qualify for the study:

• 18 years of age or older
• Received radiation therapy for head and neck cancer at least 3 years ago
• no history of recurrent cancer or a second primary cancer
• Have abnormal parotid saliva gland function (resulting in dry mouth)

*Other criteria apply

Study participants can expect the following:

• Attend 3 screening visits to confirm study qualification
• Receive an injection of the study drug into both parotid salivary glands
• Return to the study clinic for 8 follow-up visits over 1 year to monitor health and safety

Study participation will last for about one year. At the end of the AQUAx2 study, participants will be invited to enroll in a separate long-term follow-up study where active gene therapy will be given if the participant received placebo in the first year of the study. During the long term follow-up study the participants health will be monitored for up to 4 or 5 more years.